ArticleMethods in molecular biology (Clifton, N.J.)2016
Intravascular AAV9 Administration for Delivering RNA Silencing Constructs to the CNS and Periphery.
Article in Methods in molecular biology (Clifton, N.J.), 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
4 citing papers in PubMed, 6 citations in OpenAlex.
- Therapeutic AASS inhibition by AAV-miRNA rescues glutaric aciduria type I severe phenotype in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Engineering Nanoparticles for Targeted Delivery of Nucleic Acid Therapeutics in Tumor.Molecular therapy. Methods & clinical development · 2019Review
- Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.Gene therapy · 2017Article
- Article
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Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
Viral vector delivery of RNA silencing constructs, when administered into vasculature, typically results in poor central nervous system (CNS) transduction due to the inability of the vector to cross the blood-brain barrier (BBB). However, adeno-associated virus serotype 9 (AAV9) has the ability to cross the BBB and robustly transduce brain parenchyma and peripheral tissues at biologically meaningful levels when injected intravenously. Recent work by our lab has shown that this method can be used to deliver RNA silencing constructs, resulting in significant reductions in gene expression in multiple brain regions and in peripheral tissues. Here, we outline a method for delivery of AAV9 vectors expressing RNA interference (RNAi) constructs that lead to robust simultaneous transduction of mouse peripheral tissues and the CNS following a single injection into the jugular vein. Additionally, we outline methods for necropsy and immunofluorescence to detect AAV9 transduction patterns in the rodent CNS following a vascular delivery.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.